Immunodeficiency Common VariablePhase 2/3NCT07923305
Phase 2 and 3 Placebo Controlled & Long-term Extension Study of Rilzabrutinib in Adults With iCVID to Evaluate Efficacy, Safety, and Tolerability
A Phase 2/3 study testing rilzabrutinib in people with Immunodeficiency Common Variable, run by Sanofi.
Trial summary
This is a 4-part study where both Part A and Part B, which starts after the completion of Part A, are randomized (2:1), double-blinded, placebo-controlled studies, followed by an open label (Part C) and long-term extension period (Part D), phase 2 and 3 study, designed to evaluate the efficacy, safety, tolerability… Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Immunodeficiency Common Variable
- What is being tested?
- rilzabrutinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 96 participants
- Where
- No sites listed yet
- Study dates
- 9 Nov 2026to 8 Jan 2036
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants who have previously been diagnosed with CVID based on the following criteria A) Documented low serum IgG compared to age-adjusted reference ranges B) Documented low IgA and/or IgM compared to age-adjusted reference range C) Immunophenotypic findings consistent with CVID D) Absence of any other defined immunodeficiency state
- Receiving immunoglobulin IV or SC replacement therapy
- Participants who have been diagnosed with one or more of the following inflammatory complications (iCVID) A) Interstitial lung disease (ILD) B) CVID-related cytopenia for >3 months since diagnosis - thrombocytopenia or anemia C) Lymphoproliferative disease on historical CT imaging - splenomegaly or lymphoid hyperplasia
- Eastern Cooperative Oncology Group performance status Grade 2 or lower, at screening
- Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
Who cannot join
- Active pulmonary infection at screening that could account for the HRCT abnormalities
- Clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the participant or compromise the quality of the data derived from his or her participation in the study as determined by the Investigator (e.g. hepatobiliary conditions causing hepatic impairment)
- Refractory nausea or vomiting
- Chronic uncontrolled infectious diseases as determined by the Investigator
- Laboratory abnormalities at the screening visit (e.g. elevated liver enzymes)
- Recent history of substance, alcohol abuse or mental illness
- Malabsorption syndromes, active moderate-to-severe enteropathy
- Concurrent treatment with immunosuppressive or immunomodulatory drugs other than corticosteroids The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
Trial Transparency email recommended (Toll free for US & Canada) contact
About this study
Official title: Phase 2, Randomized, Double-blind, Placebo-controlled Study (Part A) Followed by a Phase 3, Randomized, Double-blind, Placebo Controlled Study (Part B) With an Open Label Extension (Part C) and Long-term Extension (Part D) to Evaluate the Efficacy, Safety and Tolerability of Rilzabrutinib in Adult Participants With Inflammatory Common Variable Immunodeficiency (iCVID)
This is a 4-part study where both Part A and Part B, which starts after the completion of Part A, are randomized (2:1), double-blinded, placebo-controlled studies, followed by an open label (Part C) and long-term extension period (Part D), phase 2 and 3 study, designed to evaluate the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of rilzabrutinib in adult patients with iCVID.
In Part A, all participants will have iCVID associated ILD and at least one additional inflammatory/ autoimmune complication (cytopenia [thrombocytopenia, anemia] and/or lymphoproliferative disease [splenomegaly, lymphoid hyperplasia]). At the completion of 24 weeks in Part A, participants will be assessed to the predefined No Response Citeria and may elect to discontinue the DB period (Part A) and enter the OL period (Part C).
Read more
Alternatively, for Part B, all participants will have CVID associated with at least one of the following: ILD, other inflammatory/ autoimmune complications (cytopenia, lymphoproliferative disease).
Part A: 32-week DB treatment of rilzabrutinib or placebo. Part B: 32-week DB treatment of rilzabrutinib or placebo. Part C: 32-week OL treatment of rilzabrutinib. Part D: OL treatment of rilzabrutinib until the last-patient-in (LPI- Part D) completes 52 weeks of treatment.
- Conditions
- Immunodeficiency Common Variable
- Being tested
- rilzabrutinib, placebo
- Medical subject headings
- Common Variable Immunodeficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rilzabrutinibDrug
Oral tablet
placeboDrug
Oral tablet
| Study group | Role | What they receive |
|---|---|---|
| rilzabrutinib | Experimental | rilzabrutinib Receives: Drug: rilzabrutinib |
| placebo | Placebo comparator | placebo Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of participants with iCVID who meet any responder definitions
In participants with ILD at baseline, a responder is defined as achieving a clinically meaningful response at Week 32.
In participants with cytopenia (thrombocytopenia, anemia) at baseline, a responder is defined as achieving therapeutic efficacy at Week 32, defined as Durable platelet response or Durable hemoglobin response (DHR) between W16 and W32, and no rescue medication for thrombocytopenia is taken at any time.
In participants with lymphoproliferative disease (splenomegaly or lymphoid hyperplasia) at baseline, a responder is defined as achieving therapeutic efficacy by Week 32.
Measured: At Week 32
Secondary outcomes (6)
Change from baseline in FACIT-Fatigue total score at Week 24 in Part A and Week 32 in Part B
Measured: At Week 24 in Part A and Week 32 in Part B
Percent change from baseline in FVC% predicted at Week 32
Measured: At Week 32
Percent change from baseline in diffusing capacity of the lung for carbon monoxide at Week 32
Measured: At Week 32
Change from baseline in FACIT-dyspnea severity score at Week 24 in Part A and Week 32 in Part B
Measured: At Week 24 in Part A and Week 32 in Part B
Change from baseline in time spend in moderate to vigorous physical activity measured with a wearable device
Measured: At Week 32
Incidence of treatment-emergent adverse events (TEAE), serious AEs (SAE), TEAE leading to permanent discontinuation, adverse events of special interest (AESI)
including clinical laboratory evaluations, vital sign measurements, and electrocardiograms
Measured: Until Week 116 of LPI for Part D
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Nov 2026
- Study start9 Nov 2026 (estimated)
- Primary completion10 May 2035 (estimated)
- Study completion8 Jan 2036 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immunodeficiency Common VariableStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 96 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07923305
- Sponsor's protocol ID
- EFC20516
- Other IDs
- 2026-525354-10, U1111-1325-2663
- Status last verified
- Nov 2026
- First submitted
- 28 Oct 2026
- Last updated
- 6 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2, Randomized, Double-blind, Placebo-controlled Study (Part A) Followed by a Phase 3, Randomized, Double-blind, Placebo Controlled Study (Part B) With an Open Label Extension (Part C) and Long-term Extension (Part D) to Evaluate the Efficacy, Safety and Tolerability of Rilzabrutinib in Adult Participants With Inflammatory Common Variable Immunodeficiency (iCVID)
- ClinicalTrials.gov ID
- NCT07923305
- Acronym / sponsor ID
- EFC20516
- Conditions
- Immunodeficiency Common Variable
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 96 participants (estimated)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 9 Nov 2026 (estimated)
- Primary completion
- 10 May 2035 (estimated)
- Study completion
- 8 Jan 2036 (estimated)
- First posted
- 6 Nov 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a 4-part study where both Part A and Part B, which starts after the completion of Part A, are randomized (2:1), double-blinded, placebo-controlled studies, followed by an open label (Part C) and long-term extension period (Part D), phase 2 and 3 study, designed to evaluate the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of rilzabrutinib in adult patients with iCVID.
In Part A, all participants will have iCVID associated ILD and at least one additional inflammatory/ autoimmune complication (cytopenia [thrombocytopenia, anemia] and/or lymphoproliferative disease [splenomegaly, lymphoid hyperplasia]). At the completion of 24 weeks in Part A, participants will be assessed to the predefined No Response Citeria and may elect to discontinue the DB period (Part A) and enter the OL period (Part C).
Alternatively, for Part B, all participants will have CVID associated with at least one of the following: ILD, other inflammatory/ autoimmune complications (cytopenia, lymphoproliferative disease).
Part A: 32-week DB treatment of rilzabrutinib or placebo. Part B: 32-week DB treatment of rilzabrutinib or placebo. Part C: 32-week OL treatment of rilzabrutinib. Part D: OL treatment of rilzabrutinib until the last-patient-in (LPI- Part D) completes 52 weeks of treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| rilzabrutinib | Experimental | Drug: rilzabrutinibrilzabrutinib |
| placebo | Placebo comparator | Drug: placeboplacebo |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Participants who have previously been diagnosed with CVID based on the following criteria A) Documented low serum IgG compared to age-adjusted reference ranges B) Documented low IgA and/or IgM compared to age-adjusted reference range C) Immunophenotypic findings consistent with CVID D) Absence of any other defined immunodeficiency state
- Receiving immunoglobulin IV or SC replacement therapy
- Participants who have been diagnosed with one or more of the following inflammatory complications (iCVID) A) Interstitial lung disease (ILD) B) CVID-related cytopenia for >3 months since diagnosis - thrombocytopenia or anemia C) Lymphoproliferative disease on historical CT imaging - splenomegaly or lymphoid hyperplasia
- Eastern Cooperative Oncology Group performance status Grade 2 or lower, at screening
- Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
Key exclusion criteria (8 listed)
- Active pulmonary infection at screening that could account for the HRCT abnormalities
- Clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the participant or compromise the quality of the data derived from his or her participation in the study as determined by the Investigator (e.g. hepatobiliary conditions causing hepatic impairment)
- Refractory nausea or vomiting
- Chronic uncontrolled infectious diseases as determined by the Investigator
- Laboratory abnormalities at the screening visit (e.g. elevated liver enzymes)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| rilzabrutinibOral tablet | Drug | rilzabrutinib |
| placeboOral tablet | Drug | placebo |
Primary outcome
Proportion of participants with iCVID who meet any responder definitions
Time frame: At Week 32
In participants with ILD at baseline, a responder is defined as achieving a clinically meaningful response at Week 32. In participants with cytopenia (thrombocytopenia, anemia) at baseline, a responder is defined as achieving therapeutic efficacy at Week 32, defined as Durable platelet response or Durable hemoglobin response (DHR) between W16 and W32, and no rescue medication for thrombocytopenia is taken at any time. In participants with lymphoproliferative disease (splenomegaly or lymphoid hyperplasia) at baseline, a responder is defined as achieving therapeutic efficacy by Week 32.
6 secondary outcomes
Change from baseline in FACIT-Fatigue total score at Week 24 in Part A and Week 32 in Part B
Time frame: At Week 24 in Part A and Week 32 in Part B
Percent change from baseline in FVC% predicted at Week 32
Time frame: At Week 32
Percent change from baseline in diffusing capacity of the lung for carbon monoxide at Week 32
Time frame: At Week 32
Change from baseline in FACIT-dyspnea severity score at Week 24 in Part A and Week 32 in Part B
Time frame: At Week 24 in Part A and Week 32 in Part B
Change from baseline in time spend in moderate to vigorous physical activity measured with a wearable device
Time frame: At Week 32
Incidence of treatment-emergent adverse events (TEAE), serious AEs (SAE), TEAE leading to permanent discontinuation, adverse events of special interest (AESI)
Time frame: Until Week 116 of LPI for Part D
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2026.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Trial Transparency email recommended (Toll free for US & Canada) contact
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing rilzabrutinib in people with Immunodeficiency Common Variable, run by Sanofi.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 6 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Trial Transparency email recommended (Toll free for US & Canada) (800-633-1610 ext. option 6, Contact-US@sanofi.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of participants with iCVID who meet any responder definitions (time frame: At Week 32). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Nov 2026; AzTrials last refreshed its copy on 6 Nov 2026.